# Notch-Modulatory Hydrogel for Dystrophic Muscle Stem Cell Rejuvenation and Expansion

> **NIH NIH R01** · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · 2022 · $29,788

## Abstract

PROJECT ABSTRACT
 Duchenne muscular dystrophy is a genetic disease caused by defects in a protein known as dystrophin.
Dystrophin functions to provide structural and mechanical stability to the muscle cells; and the absence of
functional dystrophin in Duchenne muscular dystrophy leads to progressive muscle degeneration, weakness,
and a significantly reduced life expectancy due to cardiorespiratory failure. There is no cure. As a potential
curative therapy, restoration of dystrophin through muscle stem cell transplantation is actively being explored,
but the clinical translation of these efforts is hampered by the inability to generate large quantities of muscle stem
cells in dishes without compromising their therapeutic potency. In addition, it is also very challenging to expand
muscle stem cells derived from dystrophic muscles in dishes due to their inherent functional deficits. This further
limits strategies to gene correct patient-derived muscle stem cells outside the body, grow them in a dish, and
transplant the functional muscle stem cells in an autologous manner. In this Diversity Supplement application,
we will develop a Notch-modulatory muscle stem cell culture platform that enables the generation of large
quantities of therapeutically potent muscle stem cells. This research strengthens and expands the scope of the
parent R01 project by engineering a hydrogel platform to support dystrophic muscle cell expansion for cell
therapy applications. Towards this goal, Aim 1 will engineer a Notch-modulatory hydrogel for rejuvenating
dystrophic muscle stem cells ex vivo. Aim 2 will determine the effects of suppressing p38α/β mitogen-activated
protein kinase (MAPK) activity of dystrophic muscle stem cells maintained within the Notch-modulatory hydrogel
long-term. The outcomes of this research will trailblaze a pathway towards personalized (autologous) muscle
stem cell therapy for safely treating Duchenne muscular dystrophy, where such technologies will also enable ex
vivo gene correction on the muscle stem cells outside the patient's body before being re-introduced to restore
dystrophin.

## Key facts

- **NIH application ID:** 10629737
- **Project number:** 3R01AR080616-01S1
- **Recipient organization:** ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
- **Principal Investigator:** Woojin Han
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $29,788
- **Award type:** 3
- **Project period:** 2022-03-01 → 2023-01-25

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10629737

## Citation

> US National Institutes of Health, RePORTER application 10629737, Notch-Modulatory Hydrogel for Dystrophic Muscle Stem Cell Rejuvenation and Expansion (3R01AR080616-01S1). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/10629737. Licensed CC0.

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